Priothera – US FDA grants Orphan Drug Designation to mocravimod to improve the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with hematologic malignancies
Phase 2Orphan DrugCell TherapyImmunotherapy
Mocravimod is the only S1P receptor modulator being developed as an adjunctive and maintenance treatment for blood cancers
Mocravimod is being investigated in a global registration-enabling Phase 3 trial
This is the second Orphan Drug designation for mocravimod
Priothera Ltd., a Phase 3 clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) to mocravimod for the ‘treatment to improve outcome following hematopoietic stem cell transplantation in hematologic malignancies’. This ODD aims to potentially increase leukemia-free survival by enhancing a graft-versus-leukemia (GvL) response.
Florent Gros, Co-Founder and CEO of Priothera, commented: “We are very pleased that the US FDA granted mocravimod this Orphan Drug designation. This designation emphasizes the importance of developing novel therapeutic options to improve the outcome and success of maintenance therapy following allo-HSCT in blood cancer patients. This is an important milestone as this ODD complements the first ODD granted for prevention of graft-versus-host disease.
“The two ODDs highlight mocravimod’s dual mode of action which for the first time is being leveraged to improve the allo-HSCT treatment outcomes in hematological malignancies to potentially increase the leukemia free survival – graft-versus-leukemia response - while reducing tissue damage resulting from the graft-versus-host disease.”
The first ODD granted for mocravimod by the US FDA was for the ‘prevention of graft-versus-host disease (GvHD)’ – see press release here.
Mocravimod, a sphingosine-1-phosphate (S1P) receptor modulator, is being investigated in a pivotal global Phase 3 study - MO-TRANS (NCT05429632) - evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to allo-HSCT. The study which is enrolling approximately 250 adult Acute Myeloid Leukemia (AML) patients, is ongoing in the US, Europe, Southeast Asia and Latin America.
Mocravimod, which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allo-HSCT. Moreover, it has shown a clinically meaningful outcome in a Phase 1b/2a study1 in patients with hematologic malignancies undergoing allo-HSCT.
About mocravimod
Mocravimod (also known as KRP203) is a synthetic, S1P receptor modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CAR-T cell therapy.
Mocravimod is currently being investigated as an adjunctive and maintenance treatment in a Phase 3 study for patients with AML receiving allogeneic HSCT. Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.
Priothera leverages mocravimod’s dual mode of action to maintain the beneficial graft-versus-leukemia/lymphoma (GvL) activity, while reducing tissue damage resulting from graft-versus-host disease (GvHD), both a consequence of allo-HSCT. This novel treatment approach – mocravimod being the only S1P receptor modulator treating blood cancers – tackles a high unmet medical need and aims to improve patients’ quality of life.
About Priothera
Priothera is leading the way in developing orally applied S1P receptor modulators for the treatment of hematological malignancies and for the improvement of CAR-T cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Unlike immunosuppressive drugs, mocravimod does not suppress the GvL benefits in patients receiving allogeneic HSCT while inhibiting GvHD.
Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden), EarlyBird Venture Capital (Berlin, Germany), as well as non-dilutive financing in the form of loans from the European Investment Bank under its Venture Debt Instrument and Bpifrance (Grand Est Bpifrance) in the form of a R&D innovation loan.
The content above comes from the network. if any infringement, please contact us to modify.
Organizations
Targets
-Chat with Hiro
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.